Advance cell and gene therapy trials confidently with insights from ex-regulators and experienced project leaders
Cell and gene therapies (CAGT) present new regulatory and manufacturing challenges, operational complexity, and obstacles to market and patient access. To help you navigate this complexity, we bring a team of 30+ cross-functional CAGT professionals that includes six ex-FDA regulators from the Center for Biologics Evaluation and Research (CBER)’s Office of Tissues and Advanced Therapies, the supervisory medical reviewer from one of the first approved CAR-T cell therapies, and chemistry, manufacturing, and controls (CMC) regulatory experts from EMA and MHRA.
Leverage multidisciplinary expertise with our Center of Excellence
Our Cell and Gene Therapy Center of Excellence (COE) integrates regulatory expertise with a cross-functional team to get your treatment to market faster. This team includes medical directors and regulatory affairs experts, as well as experts in clinical logistics, CMC, oncology clinical operations, data management, translational medicine, and biostatistics. They also align closely with our patient engagement team, with leadership reporting directly to our Chief Patient Officer to ensure vital patient insights are applied at every stage of development.
The Cell & Gene Center of Excellence is really a foundational element within Parexel. Seated in our patient and site engagement group, we really believe that walking the journey with our patients and with our clinical sites and sponsors in terms of how these advanced therapies are developed is a critical path to more patients being treated and better medicines being available to them.
Our mission is to live that journey with our patients and to hopefully accelerate our abilities to bring new medicines to market faster.
It's always important to understand that patient pathway from a clinical perspective. And I think as our medics are really well trained and versed in cell and gene therapies, understanding the value proposition of accelerating the trial, getting access to more patients, but also being very mindful of those key safety parameters that go on with particular gene therapy or cell therapy assets is key.
Our medics bring that to bear in each of our trials and as we go forward and learn more, we're able to share more information in real time and in a purpose-driven way such that sponsors have that type of intel to draw on and, you know, basically develop better and more lasting types of clinical designs.
Our expertise in regulatory consulting has really led the way in terms of developing cell and gene therapies at Parexel. Coupled with our commitment in operations as well as in project leadership, we understand the pathway that sponsors need to activate and navigate for developing advanced therapies. And coupled with our end-to-end performance in terms of our access group and commercialization, we believe we have a viable position in place for developing these assets faster. I would hope that sponsors and customers in general would understand that we have that investment, that commitment and passion to developing these advanced therapies.
We care deeply about their patients and our patients and how we share that knowledge in real time and create an ecosystem of partnering is really quite key to that patient and their clinical outcome.
Our experts in cell and gene therapies
Chris Learn, Ph.D., P.M.P.
Senior Vice President, Head of the Cell and Gene Center of Excellence
Steve Winitsky, M.D.
Vice President, Technical - Regulatory Strategy
Paul Bridges, Ph.D.
President, Consulting
Simona Stankeviciute, M.D., M.Sc.
Vice President, Technical
Jingke Yang, M.D., Ph.D.
Global TA Section Head, Cell & Gene Therapy, Oncology
Nancy Lunney, MA, LPA
HSP Project Director and Program Leader, Cell and Gene Therapy Center of Excellence
Rajiv Gangurde, Ph.D.
Vice President Technical - Regulatory Strategy
Christiane Niederlaender
Vice President, Technical - Regulatory Strategy
Our experts in cell and gene therapies
Chris Learn, Ph.D., P.M.P.
Senior Vice President, Head of the Cell and Gene Center of Excellence
Steve Winitsky, M.D.
Vice President, Technical - Regulatory Strategy
Paul Bridges, Ph.D.
President, Consulting
Simona Stankeviciute, M.D., M.Sc.
Vice President, Technical
Jingke Yang, M.D., Ph.D.
Global TA Section Head, Cell & Gene Therapy, Oncology
Nancy Lunney, MA, LPA
HSP Project Director and Program Leader, Cell and Gene Therapy Center of Excellence
Experience in the past 5 years with cell & gene therapies
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clinical projects
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enrolled patients
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sites
Advantages
Early advisory service
To streamline development for novel treatments, it’s vital to prepare a strong scientific argument and proof of concept before meeting with regulatory agencies. That’s why we combine an early advisory service of medical, regulatory, genomics, and biostatistical specialists with an experienced, multidisciplinary team and key technology platform partnerships — to give you a faster, smarter route to proof of concept.
Global EMR data to guide site selection
At Parexel, we use a global health research network to access EMR data for 188+ million patients worldwide. This network empowers us to identify global specialty institutions treating patients with specific ICD-10 codes, treatments, diagnoses, and procedures — so we can match sites to your essential study criteria.
Cell & Gene Therapy Training Academy
Our Cell and Gene Therapy Training Academy trains staff from across our business to improve performance across trials. Modules cover a variety of topics, including history, patient pathways, safety risks, operational fundamentals in delivering cell and gene therapy, and the study-specific content/mechanism of action/route.
Patient-focused specialists
Our expert staff in biomarker and genomic medicine, bioanalysis, modeling and simulation, and clinical pharmacology combine scientific rigor and patient-community engagement to maximize patient inclusion. We focus on high-quality delivery and understanding of study settings, driven by a skilled and flexible project management team. We listen to the voices of patients, their caregivers, and healthcare professionals, so they can confidently decide if the trial is right for them. And we ensure that the patient perspective is retained throughout the duration of the trial.
FREQUENTLY ASKED QUESTIONS
Given their novel nature and the rapid expansion of their development, CAGTs present new challenges for regulators. In many regions, CAGT regulatory frameworks have yet to be created. Where approval pathways do exist, requirements are often unclear and vary by national health authority.
To maximize the value of time spent with FDA and EMA reviewers, the Parexel team leads sponsors in preparatory mock meetings, rehearsing multiple scenarios developed by our former regulators. Parexel’s experienced consultants can also help craft arguments that will resonate with regulatory officials.
In addition, Parexel’s regulatory consultants can help distinguish between specific sections of health authority guidance documents that must be followed and sections that may not apply to a sponsor’s particular situation. For example, general guidance could recommend analysis using a specific primary efficacy endpoint or timepoint. But that recommendation might not be optimal for capturing patient benefit or even feasible based on product characteristics or the target patient subpopulation.
CAGT clinical trials present deeper levels of complexity, especially early phase trials. Our team of more than 30 cross-functional CAGT professionals understand these challenges and can help you navigate them.
Additionally, current research infrastructure is stretched, making it difficult to support the increase in CAGT clinical development. At Parexel, we are working to identify new clinics — particularly those outside of academic settings — that demonstrate potential for success. Emerging sites and investigators will require additional training and monitoring visits. But that investment will yield returns for patients and sponsors.
To streamline study startup, sponsors and CROs should grow their site networks. At Parexel, our Site Alliance Network includes more than 500 sites and 21,000 investigators.
Cell and gene therapies do not fit existing frameworks for valuing, pricing, and reimbursing pharmaceuticals. Designed to deliver a lifelong cure—or durable disease-modifying benefits—they enter a healthcare marketplace that runs on annual budgets. For payers, the presumed one-time, front-loaded cost of CAGTs has almost no precedent.
To manage potential risks and controversies, sponsors can strengthen their scientific and clinical validation of novel endpoints by collecting patient-reported outcome (PRO) data and quantifying a range of medical and economic cost-offsets in their evidence dossier.
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Podcast: Accelerated approvals – what’s next, confirmatory trials, access, and pricing
Aug 5, 2024
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Enabling Successful Sites, Episode 2: Empowering Sites to be More Inclusive Through Cultural Sensitivity Training
Jun 28, 2024
Blog
Making cell and gene therapy more accessible in the treatment of solid tumors
Jun 4, 2024
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May 28, 2024
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May 15, 2024
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May 7, 2024
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Mar 6, 2024
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Feb 27, 2024
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CAR-T boxed warnings: regulatory precedents and opportunities
Feb 9, 2024
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Jan 31, 2024
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Jan 10, 2024
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May 22, 2023
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Mar 28, 2023
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Jan 12, 2023
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Jul 22, 2022
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Apr 4, 2022
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